Ruxoprubart

Following achievements in Asia, Roche's PNH treatment drug PIASKY (crovalimab) received its first FDA approval. This approval has spiked the competition between Roche and current market leader AstraZeneca. Additionally, the other pharma companies also geared up to launch their respective lead assets. LAS VEGAS, Sept. 2, 2024 /PRNewswire/ -- Paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad of features: intravascular hemolysis, thromboembolic events, and cytopenia. Manifestations vary among patients, making classification based on typical presentations challenging due to the disease's unpredictable nature. In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were ~12,000 cases in the 7MM, which might reach ~13,000 cases by 2034. In the 7MM, the highest number of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were observed in the US. Until 2007, PNH was a devastating disease without treatment for hemolysis and thrombosis, the leading cause of death in patients. However, the last decade saw a revolutionary shift with the advent of the anti-C5 agent eculizumab. It significantly reduced hemolysis, decreased transfusion dependency, and, crucially, lowered the thrombosis rate. The disease-modifying therapeutic strategy for paroxysmal nocturnal hemoglobinuria includes complement inhibition therapy, with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by the FDA, and considered the gold standard. SOLIRIS, the pioneering therapy for PNH, was succeeded by ULTOMIRIS, both developed by Alexion Pharmaceuticals, offering C5 inhibitors as essential therapeutic options. Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to offer improved treatment with fewer side effects. Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on findings from the global Phase III COMMODORE 2 clinical trial. The FDA accepted a biologics license application for crovalimab in September 2023 and subsequently approved it in June 2024 for treating PNH in adults and adolescents aged 13 years and older. Genentech, a subsidiary of Roche, has made the drug available in the US. In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and above. In February 2024, China's National Medical Products Administration (NMPA) approved crovalimab for treating PNH in adults and adolescents aged 12 years and older who have not previously received complement inhibitor treatment, following a priority review that began in August 2022. PiaSky is also undergoing review by other regulatory authorities, including those in Europe and Taiwan. To know more about paroxysmal nocturnal hemoglobinuria treatment options, visit @ New Treatment for Paroxysmal Nocturnal Hemoglobinuria The paroxysmal nocturnal hemoglobinuria market is crowded with so many companies working in the domain. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran, OMS906, NM8074 (ruxoprubart), and others, reflecting a dynamic evolution in treatment strategies. Discover which therapies are expected to grab major paroxysmal nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report Pozelimab is a novel, fully human monoclonal antibody currently under investigation. It aims to inhibit complement factor C5, thereby preventing the destruction of red blood cells (hemolysis) responsible for symptoms in conditions like Paroxysmal Nocturnal Haemoglobinuria (PNH) and other diseases influenced by complement pathway activity. This IgG4 antibody binds tightly to both wild-type and variant forms of human C5, effectively blocking its function. In its ongoing development, pozelimab is also being studied in combination with Alnylam's cemdisiran, a siRNA C5 inhibitor. This investigational combination therapy is aimed at treating various complement-mediated disorders, including PNH and myasthenia gravis. OMS-906 is currently being developed to treat paroxysmal nocturnal hemoglobinuria (PNH), complement 3 glomerulopathy (C3G), idiopathic immune complex-mediated glomerulonephritis (ICGN), and arthritis. It can be administered either subcutaneously or intravenously. This drug candidate is a monoclonal antibody designed to target mannan-binding lectin serine protease 3 (MASP-3) to exert its therapeutic effects. Discover more about drugs for paroxysmal nocturnal hemoglobinuria in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials The anticipated launch of these emerging therapies for paroxysmal nocturnal hemoglobinuria are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the paroxysmal nocturnal hemoglobinuria market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that in 2023, the total paroxysmal nocturnal hemoglobinuria market size in the 7MM was USD ~1.4 billion, which is expected to reach USD ~2.5 billion by 2034. This growth can be attributed to the advances in disease mechanisms that drive new diagnostics and therapeutics, fueling drug development. Additionally, the PNH market foresees positive growth, propelled by increasing cases and upcoming therapies in the forecast period. DelveInsight's latest published market report titled as Paroxysmal Nocturnal Hemoglobinuria Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the paroxysmal nocturnal hemoglobinuria country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of PNH Gender-specific Cases of PNH The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM paroxysmal nocturnal hemoglobinuria market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this paroxysmal nocturnal hemoglobinuria market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the paroxysmal nocturnal hemoglobinuria market. Also, stay abreast of the mitigating factors to improve your market position in the paroxysmal nocturnal hemoglobinuria therapeutic space. Related Reports Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted paroxysmal nocturnal hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

-- The United States FDA Clears Initiation of Efficacy Trial in Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV) Patients APhase II, Efficacy Trial in adult AAV patients has been approved by the FDA. Ruxoprubart is a first-in-class treatment for sustaining remission in AAV patients as a conjunctive therapy along with Standard of Care (SOC) which includes corticosteroids plus Cyclophosphamide/Azathioprine or Rituximab. Ruxoprubart demonstrated an anticipated safety pro Phase I trial in healthy subjects in 2022. TAVNEOS® (avacopan) was approved by the FDA in 2022 for treating AAV. Ruxoprubart is being positioned as the next-generation AAV treatment based on its mechanism of action. NovelMed’s Ruxoprubart drug blocks the alternative pathway (AP) without blocking the complement classical pathway (CP), an important advantage in maintaining a healthy lifestyle. Recent commencement of Ruxoprubart proof-of-concept Phase II trial through intravenous (IV) administration in naïve PNH patients shows promising results with a clean safety and efficacy profile. The Subcutaneous (SC) route of administration is under regulatory review and is expected to start soon. Ruxoprubart is a multi-indication platform. Given the role of the alternative pathway in rare diseases, the antibody drug has the potential to treat multiple diseases, including ocular, inflammatory, renal, neurological, and hematological. CLEVELAND, June 17, 2024 (GLOBE NEWSWIRE) -- NovelMed Therapeutics, Inc. proudly announces the U.S. Food and Drug Administration's (FDA) clearance on Ruxoprubart, the investigational drug, for commencing an efficacy trial targeting patients with ANCA Associated Vasculitis (AAV), a rare autoimmune chronic ailment characterized by inflammation in small blood vessels. Currently, the standard of care (SOC) for AAV requires high doses of Glucocorticoids, along with prolonged administration of other immunosuppressive drugs. However, this approach comes with severe side effects, including general immune suppression and toxicity from glucocorticoids, leaving unmet needs in managing the disease's impact on vital organs and associated adverse events (AEs). Results from Phase I and II trials of Ruxoprubart in PNH patients demonstrate the drug's selective blocking of the alternative pathway (AP) without affecting the classical pathway (CP), which is crucial for maintaining immunity. This selectivity presents a significant advantage over existing complement blockers approved by the FDA or under development, potentially eliminating the need for a Black Box Warning. In a forthcoming twelve-patient AAV study, Ruxoprubart will be evaluated alongside the SOC, akin to the trial conducted for TAVNEOS. Ruxoprubart is anticipated to outperform TAVNEOS due to its comprehensive control over the pathway responsible for generating three major toxins contributing to the disease pathology: C3a, C5a, and MAC. While TAVNEOS only targets C5a, Ruxoprubart aims to neutralize two powerful toxins including C3a and C5a, offering a potentially superior treatment approach. "We are thrilled to have gained FDA approval to address AAV in patients with an inadequate response to standard of care. Through this study, we aim to compare Ruxoprubart with historical data on TAVNEOS, hoping to improve AAV patients' lives substantially" says Dr. Rekha Bansal, Chief Executive Officer of NovelMed. Joseph Jankowski, Ph.D., VP, Business Development of NovelMed, expressed enthusiasm regarding the FDA’s clearance, stating, "The FDA's clearance of the Phase II clinical trial for AAV marks a significant milestone for Ruxoprubart, which harbors potential as a versatile platform for multiple indications." Ruxoprubart is positioned to compete with TAVNEOS with its potential for a superior safety and efficacy profile. Acquisition of TAVNEOS by Amgen for 3.7 Billion dollars underscores the value placed on treatments for AAV patients. Ruxoprubart is expected to be a next-generation product that can replace TAVNEOS. Ruxoprubart's selectivity and specificity, based on the clinical data from ongoing trials, hold promise not only for AAV, but also for several other renal disorders, including aHUS, and IgAN. The drug's potential to offer a superior safety and efficacy pro it apart and places it in a strong position within the market. The alternative pathway (AP) plays a pivotal role in managing anemia, inflammation, and tissue damage in various complement-mediated disorders, including AAV. NovelMed is committed to advancing the development of Ruxoprubart and addressing the unmet medical needs of patients with AAV and other complement-mediated disorders. The FDA's clearance of the Phase II trial represents a significant step forward in our mission to bring innovative and effective treatments to the market. We are actively exploring licensing, partnership, and acquisition opportunities to advance Ruxoprubart's development and approval across multiple rare disease indications. For further details, please visit . About ANCA Associated Vasculitis (AAV) Anti-Neutrophil Cytoplasmic Antibody-associated vasculitis (ANCA Vasculitis or AAV), is a rare autoimmune condition causing inflammation in blood vessels, often leading to swelling, narrowing, and sometimes blockage of blood vessels. It is characterized by the presence of Anti-Neutrophil Cytoplasmic Antibodies (ANCAs), targeting certain proteins within white blood cells, particularly neutrophils. It often causes a specific type of inflammation called crescentic glomerulonephritis, which can lead to kidney failure. Diagnosis of AAV involves a comprehensive assessment of medical history, physical examination, specific laboratory tests, imaging studies, and sometimes tissue biopsies. The current treatment approach primarily focuses on immune system suppression to alleviate inflammation and prevent further organ damage, utilizing corticosteroids and immunosuppressants. Despite being a rare and complex disease, advancements in treatment strategies are a must for continued improvement in the outcome of AAV patients. The prevalence of AAV in the United States is approximately 66,600 to 133,200 patients. TAVNEOS costs $75,051 annually per patient, with the potential to generate over 4 Billion in annual sales. About Ruxoprubart Ruxoprubart is a high affinity, humanized monoclonal antibody to Bb, selectively blocking the initiation and propagation of the alternative pathway (AP). By targeting the AP and sparing the CP pathway, Ruxoprubart holds promise for treating renal disorders, including ANCA vasculitis (AAV). Furthermore, Ruxoprubart shows therapeutic potential across a broad spectrum of rare diseases and disorders, including but not limited to hematological (PNH), renal (aHUS, IgAN), neurological (NMOSD), and ocular (GA and Uveitis). Clinical trials for Ruxoprubart can be found at , showing its potential to address various complement-mediated and associated illnesses where treatment options are inadequate or absent. About NovelMed NovelMed is a clinical-stage biopharmaceutical company dedicated to developing antibody treatments for numerous complement-mediated disorders, particularly rare (orphan) diseases. We aim to innovate and develop novel biologics to address unmet needs in rare disease treatment. NovelMed boasts a robust portfolio of intellectual property with broad applications in rare and common diseases. This includes Ruxoprubart's potential to treat a range of complement-mediated disorders associated with uncontrolled AP activation. As part of its antibody platform, NovelMed also offers humanized antibodies to C3b and Factor P (Properdin), effectively targeting the AP. NovelMed actively seeks licensing, partnership, and acquisition opportunities to advance antibody development through Phase III and approval in multiple rare disease indications. For more information, please visit News (novelmed.com). Ya Gao Email: bd@novelmed.com Tel: (216) 440 2696 Follow NovelMed on LinkedIn